Alnylam Maps Out First Steps in ‘RNA Decade’

Xconomy Boston — 

Alnylam Pharmaceuticals CEO John Maraganore had a snappy greeting ready for our first conversation of 2010.

“Happy RNA Decade,” Maraganore said.

For the sake of his Cambridge, MA-based company (NASDAQ: ALNY), Maraganore is wagering that this will be the decade in which scientific seeds of RNA-based therapies start to fulfill their potential. The decade-long vision is that RNA interference, microRNA therapies, RNA activation, and other treatments like them will emerge as new classes of therapy that can hit specific molecular targets, and regulate disease processes that targeted antibody drugs or conventional small-molecule chemicals can’t. And, oh yes, Alnylam is seeking to be one of the trailblazers of RNA, if not the dominant player.

While Maraganore was clearly in the mood to think big, and long-term, about RNA-based technology when we talked, he knows that the biotech investment world that gathers today at the JP Morgan Healthcare Conference in San Francisco is more interested in what Alnylam is going to do this quarter, and this year, rather than the rest of the decade. So we talked about both the short term and long term.

To keep things in perspective, it’s been a little more than a decade since the discovery of RNA interference, which can turn off specific genes. That discovery has given birth to new ideas on how to turn on desirable genes (RNA activation), ways to turn off whole biological networks (microRNA), and ways to regulate long-non coding RNA. While those technologies have excited researchers in the lab and produced reams of scientific papers, no RNAi drug has yet navigated the long, difficult journey to become an FDA-approved product.

But this will be the decade that sort of progress materializes, Maraganore predicts.

“This feels a lot to me like the ’90s,” Maraganore says. “Most people would agree looking back that the ’90s were the antibody decade. I would say this is going to be the RNA decade.”

It’s a bold statement, given that history says new pharmaceutical technologies tend to boom, then bust, before they finally reach their potential and boom again. The technique to engineer … Next Page »

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